EA201992001A1 - COMPOSITIONS AND METHODS FOR INCREASING GENE EXPRESSION - Google Patents

COMPOSITIONS AND METHODS FOR INCREASING GENE EXPRESSION

Info

Publication number
EA201992001A1
EA201992001A1 EA201992001A EA201992001A EA201992001A1 EA 201992001 A1 EA201992001 A1 EA 201992001A1 EA 201992001 A EA201992001 A EA 201992001A EA 201992001 A EA201992001 A EA 201992001A EA 201992001 A1 EA201992001 A1 EA 201992001A1
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
gene expression
compositions
increasing gene
increasing
Prior art date
Application number
EA201992001A
Other languages
Russian (ru)
Inventor
Аннахита Керавала
Original Assignee
Адверум Байотекнолоджис, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Адверум Байотекнолоджис, Инк. filed Critical Адверум Байотекнолоджис, Инк.
Publication of EA201992001A1 publication Critical patent/EA201992001A1/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

В настоящем изобретении предложены полинуклеотидные кассеты, векторы экспрессии и способы экспрессии гена в клетках млекопитающих.The present invention provides polynucleotide cassettes, expression vectors and methods for gene expression in mammalian cells.

EA201992001A 2017-03-17 2018-03-16 COMPOSITIONS AND METHODS FOR INCREASING GENE EXPRESSION EA201992001A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762472892P 2017-03-17 2017-03-17
PCT/US2018/022996 WO2018170473A1 (en) 2017-03-17 2018-03-16 Compositions and methods for enhanced gene expression

Publications (1)

Publication Number Publication Date
EA201992001A1 true EA201992001A1 (en) 2020-01-24

Family

ID=63523675

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201992001A EA201992001A1 (en) 2017-03-17 2018-03-16 COMPOSITIONS AND METHODS FOR INCREASING GENE EXPRESSION

Country Status (13)

Country Link
US (3) US11773406B2 (en)
EP (1) EP3596213A4 (en)
JP (2) JP7343903B2 (en)
KR (1) KR102616820B1 (en)
CN (1) CN110546257B (en)
AU (1) AU2018234918B2 (en)
BR (1) BR112019019158A2 (en)
CA (1) CA3054942A1 (en)
EA (1) EA201992001A1 (en)
IL (1) IL268788A (en)
MX (1) MX2019011040A (en)
SG (1) SG11201907653QA (en)
WO (1) WO2018170473A1 (en)

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GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
MX2019011040A (en) 2017-03-17 2020-01-20 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression.
CN112585267A (en) * 2018-08-02 2021-03-30 诺维信公司 Preparation of a combinatorial library of DNA constructs Using introns
WO2020180951A1 (en) 2019-03-04 2020-09-10 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
US20210100856A1 (en) * 2019-09-11 2021-04-08 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
JP2022547541A (en) 2019-09-11 2022-11-14 アドヴェラム バイオテクノロジーズ， インコーポレイテッド Methods of treating intraocular neovascular diseases using AAV2 variants encoding aflibercept
BR112022003206A2 (en) * 2019-09-11 2022-08-16 Adverum Biotechnologies Inc METHODS TO TREAT NEOVASCULAR EYE DISEASES USING AAV2 VARIANTS ENCODING AFLIBERCEPTE
KR102346159B1 (en) * 2020-02-20 2022-01-03 국립암센터 High Efficiency Expression Vector and Uses thereof
US20220010332A1 (en) * 2020-07-08 2022-01-13 Neuracle Genetics Inc. Intron fragments
CN113817775B (en) * 2020-08-25 2023-02-17 南京吉迈生物技术有限公司 Modified aflibercept, compositions, methods and uses thereof in gene therapy
EP4288064A2 (en) * 2021-02-02 2023-12-13 Allen Institute Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes
JP2024515459A (en) 2021-04-27 2024-04-10 アドヴェラム バイオテクノロジーズ， インコーポレイテッド Methods of Treating Ocular Diseases Using AAV2 Variants Encoding Aflibercept
WO2023283649A1 (en) * 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
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WO2023150566A1 (en) 2022-02-02 2023-08-10 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
WO2023158990A1 (en) 2022-02-16 2023-08-24 Adverum Biotechnologies, Inc. Method of reducing cst fluctuation in neovascular amd by a recombinant adeno-associated virus
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Also Published As

Publication number Publication date
CN110546257A (en) 2019-12-06
WO2018170473A1 (en) 2018-09-20
US11352644B2 (en) 2022-06-07
JP2020513792A (en) 2020-05-21
MX2019011040A (en) 2020-01-20
NZ756504A (en) 2023-11-24
IL268788A (en) 2019-10-31
AU2018234918B2 (en) 2023-11-02
SG11201907653QA (en) 2019-09-27
CN110546257B (en) 2024-03-01
EP3596213A4 (en) 2021-02-17
BR112019019158A2 (en) 2020-05-05
CA3054942A1 (en) 2018-09-20
US20230257777A1 (en) 2023-08-17
JP7360208B2 (en) 2023-10-12
KR20190132626A (en) 2019-11-28
JP2022092057A (en) 2022-06-21
JP7343903B2 (en) 2023-09-13
US20210040501A1 (en) 2021-02-11
AU2018234918A1 (en) 2019-09-12
KR102616820B1 (en) 2023-12-21
EP3596213A1 (en) 2020-01-22
US20200010851A1 (en) 2020-01-09
US11773406B2 (en) 2023-10-03

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